The Pathway for New Cancer Drugs

Sylvester Comprehensive Cancer Center’s relationship with the FDA helps researchers navigate the lengthy approval process for new cancer therapies.

With hundreds of clinical trials currently underway, translating research breakthroughs into more effective cancer treatments remains one of the highest priorities at Sylvester Comprehensive Cancer Center, part of UHealth — University of Miami Health System.

These trials provide Sylvester’s patients with advanced access to new drugs that may eventually get approved by the U.S. Food and Drug Administration (FDA) and become the standard of care.

“We are conducting the research today that is going to become the standard for tomorrow,” said Mikkael Sekeres, M.D., M.S., chief of the Division of Hematology at Sylvester and professor of medicine at the Miller School.

Cancer Clinical Trail Design

Sylvester has a long history of working closely with the FDA.

Sylvester Comprehensive Cancer Center physician Mikkael Sekeres, M.D., M.S.
Sylvester’s Mikkael Sekeres, M.D., M.S., says patients benefit from the Center’s relationship with the FDA.

“We work hand in hand with the FDA to develop the guidelines that determine a drug’s effectiveness for a given cancer to ensure our clinical trials define efficacy the same way the FDA will define it for approval,” said Dr. Sekeres, who served on the FDA’s Oncologic Drugs Advisory Committee, the penultimate step before the agency approves a therapy, for five years and as chair for two of those years. “If the drugs are approved by the FDA, our patients often benefit from them years before they would otherwise.”

Dr. Sekeres references a new blood cancer drug to treat myelodysplastic syndromes. He was an investigator and on the steering committee for the clinical trial, and presented data at international conferences that should soon be published in a major journal.

Dr. Sekeres believes the drug will receive FDA approval in 2024.

Lengthy Drug Approval Process

The FDA’s origin as a federal consumer protection agency began with the passage of the 1906 Pure Food and Drugs Act. Until then, drugmakers could claim almost anything. With current guidelines requiring that drugs are both safe and effective, the FDA has become one of the most trusted regulatory agencies in the world.

While new drugs have contributed to significant advancements in cancer care and improved the lives of many patients, the development and approval process is lengthy. It takes an average of 10 years from discovery to the marketplace, including preclinical research (three to five years) and three phases of clinical trials on humans (three to seven years) before drugs can be submitted to the FDA for approval and post-market surveillance.

Dr. Sekeres signing his book at the Miami Book Fair.
Dr. Sekeres wrote a book analyzing the pivotal role of the FDA in the drug approval process.

Many cancer drugs have benefited from the FDA’s Accelerated Approval Program that allows drugs that fill an unmet medical need to be approved based on a surrogate endpoint and requires manufacturers to follow up with confirmation of its clinical benefit. While this program has had a positive impact on many cancer drugs, some have proven to be ineffective and removed from the market. 

In 2011, for example, the FDA announced that the breast cancer indication for Avastin (bevacizumab) was withdrawn after conclusions that the drug was not safe and effective for the treatment of breast cancer. This was one of the many topics discussed in Dr. Sekeres’ book, Drugs and the FDA: Safety, Efficacy, and the Public’s Trust.

The Goal: Improved Quality of Life for Cancer Patients

Dr. Sekeres believes that the future of drug development looks bright for cancer patients.

“We are getting more sophisticated at defining subtypes of cancer, identifying drugs that work in those subtypes of cancer, and getting approvals for specific subtypes of cancer,” he said. “We are also getting better at identifying patients who will truly benefit from a drug, as well as those who won’t, so we don’t waste their time and expose them to side effects of the drug by giving them something that won’t work.”

Dr. Sekeres recently chaired a workshop sponsored by the FDA, the American Association of Cancer Researchers and the American Statistical Association to examine the challenges of measuring overall survival in clinical trials and discuss the importance of defining clinical trial endpoints that truly reflect improved survival.

He wants to ensure that endpoints are meaningful for patients and says success for an individual patient might be living long enough to attend a wedding, while success for a pharmaceutical company might be selling more drugs.

“I consider a drug successful when it allows my patients to spend more days, weeks or months on this earth with a great quality of life,” said Dr. Sekeres.

Tags: clinical trials, Dr. Mikkael Sekeres, FDA, Sylvester Comprehensive Cancer Center